final 2 Flashcards
It is a critical step ni the drug design process. It involves identifying the molecular structures or biological pathways that are involved in a disease.
These targets are typically proteins, genes, or RNA molecules that play a crucial role in the pathology of the disease.
Target Identification
It involves identifying the drugs that are effective in treating a particular backward and then working backward to identify the molecular targets that they interact with.
Target-based pharmacology
This involves using computational methods to analyze large datasets of genetic and biological information to identify potential drug targets.
Computational/bioinformatics
This involves testing large numbers of molecules or compounds to identify those that have a specific effect on the disease target.
High-throughput screening
These rely on existing knowledge of the disease and its underlying mechanisms to identify potential drug targets.
Knowledge-based approaches
The identification of genes that are associated with a disease can provide clues to potential drug targets.
This can involve sequencing the genomes of affected individuals or studying genetic mutations that are linked to the disease.
Genetics
Using technologies like CRISPR/Cas9 or RNA interference (RNAi) to delete or reduce the expression of the target gene in cell lines or animal models.
Observing the resulting phenotype helps determine the role of the gene in the disease.
Gene Knockout/Knockdown Studies
Increasing the expression of the target gene to assess its effects on disease progression.
Overexpression Studies
Examine the biochemical activities of the target protein, such as enzyme activity, binding properties, and interaction with other molecules, to understand its role in the disease
Protein Function Studies
Assessing the binding of potential drug molecules to the target protein to confirm that modulating this interaction can have therapeutic effects
Ligand Binding Assays
Observing changes in cell behavior (e.g., proliferation, apoptosis, migration)
upon modulation of the target using small molecules, antibodies, or genetic tools.
Cellular Phenotype Assays
Studying the impact of target modulation on cellular signaling pathways involved in the disease
Pathway Analysis
Using animal models that replicate human disease conditions to study the effects of target modulation.
Successful outcomes in these models provide strong evidence for target relevance.
Disease Models
Administering compounds that modulate the target in animal models to observe therapeutic effects and potential side effects.
Pharmacological Studies
Identifying and validating biomarkers that correlate with target modulation and disease outcomes in clinical samples.
Biomarker Studies
Integrating data from preclinical studies with clinical observations to confirm the target’s role in human disease.
Translational Research
Developing a new drug can take between 8-12 years and cost over £1 billion. This includes extensive research, clinical trials, and regulatory approvals.
Length and Cost
Developing a new drug can take between __ years and cost over __.
8-12
£1 billion
The process is highly complex and uncertain. Only a small fraction of compounds that enter pre-clinical trials make it to human testing, and even fewer get approved.
Complexity and Uncertainty
For many diseases, especially neurological disorders, the underlying biological mechanisms are not fully understood, making it difficult to identify suitable drug targets.
Unknown Pathophysiology
Animal models often do not accurately replicate human diseases, leading to potential failures in later stages of development.
Limitations of Animal Models
The genetic and biological diversity among patients means that a one-size-fits-all approach is often ineffective.
Heterogeneity of Patient Populations
Navigating regulatory requirements and managing financial risks are significant challenges.
Regulatory bodies have stringent requirements that must be met, which can be time-consuming and costly.
Regulatory and Financial Pressures
Effective drug development requires scientific expertise and strong business and management skills to navigate the industrial and regulatory landscape.
Business and Management Skills
